CA3133226A1 - Procede ameliore d'integration de constructions d'adn a l'aide d'endonucleases guidees par arn - Google Patents

Procede ameliore d'integration de constructions d'adn a l'aide d'endonucleases guidees par arn

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Publication number
CA3133226A1
CA3133226A1 CA3133226A CA3133226A CA3133226A1 CA 3133226 A1 CA3133226 A1 CA 3133226A1 CA 3133226 A CA3133226 A CA 3133226A CA 3133226 A CA3133226 A CA 3133226A CA 3133226 A1 CA3133226 A1 CA 3133226A1
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Canada
Prior art keywords
donor
nucleotides
cells
seq
donor dna
Prior art date
Legal status (The legal status is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the status listed.)
Pending
Application number
CA3133226A
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English (en)
Inventor
Beibei Ding
Wenzhong Guo
Yanliang Zhang
Current Assignee (The listed assignees may be inaccurate. Google has not performed a legal analysis and makes no representation or warranty as to the accuracy of the list.)
Sorrento Therapeutics Inc
Original Assignee
Sorrento Therapeutics Inc
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Application filed by Sorrento Therapeutics Inc filed Critical Sorrento Therapeutics Inc
Publication of CA3133226A1 publication Critical patent/CA3133226A1/fr
Pending legal-status Critical Current

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    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09—Recombinant DNA-technology
    • C12N15/87—Introduction of foreign genetic material using processes not otherwise provided for, e.g. co-transformation
    • C12N15/90—Stable introduction of foreign DNA into chromosome
    • C12N15/902—Stable introduction of foreign DNA into chromosome using homologous recombination
    • C12N15/907—Stable introduction of foreign DNA into chromosome using homologous recombination in mammalian cells
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K39/00—Medicinal preparations containing antigens or antibodies
    • A61K39/46—Cellular immunotherapy
    • A61K39/461—Cellular immunotherapy characterised by the cell type used
    • A61K39/4611—T-cells, e.g. tumor infiltrating lymphocytes [TIL], lymphokine-activated killer cells [LAK] or regulatory T cells [Treg]
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K39/00—Medicinal preparations containing antigens or antibodies
    • A61K39/46—Cellular immunotherapy
    • A61K39/463—Cellular immunotherapy characterised by recombinant expression
    • A61K39/4631—Chimeric Antigen Receptors [CAR]
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K39/00—Medicinal preparations containing antigens or antibodies
    • A61K39/46—Cellular immunotherapy
    • A61K39/464—Cellular immunotherapy characterised by the antigen targeted or presented
    • A61K39/4643—Vertebrate antigens
    • A61K39/4644—Cancer antigens
    • A61K39/464402—Receptors, cell surface antigens or cell surface determinants
    • A61K39/464411—Immunoglobulin superfamily
    • A61K39/464412—CD19 or B4
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K39/00—Medicinal preparations containing antigens or antibodies
    • A61K39/46—Cellular immunotherapy
    • A61K39/464—Cellular immunotherapy characterised by the antigen targeted or presented
    • A61K39/4643—Vertebrate antigens
    • A61K39/4644—Cancer antigens
    • A61K39/464402—Receptors, cell surface antigens or cell surface determinants
    • A61K39/464416—Receptors for cytokines
    • A61K39/464417—Receptors for tumor necrosis factors [TNF], e.g. lymphotoxin receptor [LTR], CD30
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K39/00—Medicinal preparations containing antigens or antibodies
    • A61K39/46—Cellular immunotherapy
    • A61K39/464—Cellular immunotherapy characterised by the antigen targeted or presented
    • A61K39/4643—Vertebrate antigens
    • A61K39/4644—Cancer antigens
    • A61K39/464402—Receptors, cell surface antigens or cell surface determinants
    • A61K39/464424—CD20
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K39/00—Medicinal preparations containing antigens or antibodies
    • A61K39/46—Cellular immunotherapy
    • A61K39/464—Cellular immunotherapy characterised by the antigen targeted or presented
    • A61K39/4643—Vertebrate antigens
    • A61K39/4644—Cancer antigens
    • A61K39/464402—Receptors, cell surface antigens or cell surface determinants
    • A61K39/464426—CD38 not IgG
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K39/00—Medicinal preparations containing antigens or antibodies
    • A61K39/46—Cellular immunotherapy
    • A61K39/464—Cellular immunotherapy characterised by the antigen targeted or presented
    • A61K39/4643—Vertebrate antigens
    • A61K39/4644—Cancer antigens
    • A61K39/46448—Cancer antigens from embryonic or fetal origin
    • A61K39/464482—Carcinoembryonic antigen [CEA]
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09—Recombinant DNA-technology
    • C12N15/10—Processes for the isolation, preparation or purification of DNA or RNA
    • C12N15/102—Mutagenizing nucleic acids
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    • C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09—Recombinant DNA-technology
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    • C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09—Recombinant DNA-technology
    • C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/113—Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides; Antisense DNA or RNA; Triplex- forming oligonucleotides; Catalytic nucleic acids, e.g. ribozymes; Nucleic acids used in co-suppression or gene silencing
    • C12N15/1138—Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides; Antisense DNA or RNA; Triplex- forming oligonucleotides; Catalytic nucleic acids, e.g. ribozymes; Nucleic acids used in co-suppression or gene silencing against receptors or cell surface proteins
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    • C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09—Recombinant DNA-technology
    • C12N15/87—Introduction of foreign genetic material using processes not otherwise provided for, e.g. co-transformation
    • C12N15/90—Stable introduction of foreign DNA into chromosome
    • C—CHEMISTRY; METALLURGY
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    • C12N5/00—Undifferentiated human, animal or plant cells, e.g. cell lines; Tissues; Cultivation or maintenance thereof; Culture media therefor
    • C12N5/06—Animal cells or tissues; Human cells or tissues
    • C12N5/0602—Vertebrate cells
    • C12N5/0634—Cells from the blood or the immune system
    • C12N5/0636—T lymphocytes
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • C12N9/00—Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
    • C12N9/14—Hydrolases (3)
    • C12N9/16—Hydrolases (3) acting on ester bonds (3.1)
    • C12N9/22—Ribonucleases RNAses, DNAses
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/10—Type of nucleic acid
    • C12N2310/20—Type of nucleic acid involving clustered regularly interspaced short palindromic repeats [CRISPRs]
    • C—CHEMISTRY; METALLURGY
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/31—Chemical structure of the backbone
    • C12N2310/315—Phosphorothioates
    • C—CHEMISTRY; METALLURGY
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/32—Chemical structure of the sugar
    • C12N2310/321—2'-O-R Modification
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    • C12N2501/00—Active agents used in cell culture processes, e.g. differentation
    • C12N2501/70—Enzymes
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    • C12N2510/00—Genetically modified cells

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  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Genetics & Genomics (AREA)
  • Microbiology (AREA)
  • General Health & Medical Sciences (AREA)
  • Biomedical Technology (AREA)
  • Immunology (AREA)
  • Cell Biology (AREA)
  • Organic Chemistry (AREA)
  • Zoology (AREA)
  • Wood Science & Technology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Mycology (AREA)
  • Medicinal Chemistry (AREA)
  • Biotechnology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Epidemiology (AREA)
  • Veterinary Medicine (AREA)
  • Public Health (AREA)
  • Animal Behavior & Ethology (AREA)
  • General Engineering & Computer Science (AREA)
  • Molecular Biology (AREA)
  • Oncology (AREA)
  • Biochemistry (AREA)
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  • Biophysics (AREA)
  • Plant Pathology (AREA)
  • Hematology (AREA)
  • Gynecology & Obstetrics (AREA)
  • Pregnancy & Childbirth (AREA)
  • Reproductive Health (AREA)
  • Crystallography & Structural Chemistry (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)

Abstract

Un procédé amélioré, plus sûr et commercialement efficace pour développer des cellules génétiquement modifiées, est divulgué. Plus spécifiquement, un procédé comprenant l'introduction d'une construction d'ADN donneur, d'un ARN guide et d'une nucléase guidée par ARN avec les cellules hôtes à transfecter, et l'introduction des trois composants dans la cellule hôte, est divulgué. Une construction d'ADN donneur conçue pour insérer un CAR (récepteur antigénique chimérique) dans un site génomique défini d'une cellule hôte, est également divulguée. En outre, la présente divulgation concerne une cellule hôte transfectée avec un CAR exempt de vecteurs viraux pouvant poser un problème de sécurité. La divulgation concerne un procédé plus efficace et plus rentable pour l'ingénierie de cellules T pour exprimer des constructions CAR.
CA3133226A 2019-03-11 2020-03-11 Procede ameliore d'integration de constructions d'adn a l'aide d'endonucleases guidees par arn Pending CA3133226A1 (fr)

Applications Claiming Priority (5)

Application Number Priority Date Filing Date Title
US201962816836P 2019-03-11 2019-03-11
US62/816,836 2019-03-11
US201962901735P 2019-09-17 2019-09-17
US62/901,735 2019-09-17
PCT/US2020/022056 WO2020185867A1 (fr) 2019-03-11 2020-03-11 Procédé amélioré d'intégration de constructions d'adn à l'aide d'endonucléases guidées par arn

Publications (1)

Publication Number Publication Date
CA3133226A1 true CA3133226A1 (fr) 2020-09-17

Family

ID=70190148

Family Applications (1)

Application Number Title Priority Date Filing Date
CA3133226A Pending CA3133226A1 (fr) 2019-03-11 2020-03-11 Procede ameliore d'integration de constructions d'adn a l'aide d'endonucleases guidees par arn

Country Status (11)

Country Link
US (1) US20220145333A1 (fr)
EP (1) EP3938510A1 (fr)
JP (1) JP2022524435A (fr)
KR (1) KR20210149734A (fr)
CN (1) CN113825834A (fr)
AU (1) AU2020239050A1 (fr)
CA (1) CA3133226A1 (fr)
IL (1) IL286244A (fr)
MX (1) MX2021010938A (fr)
SG (1) SG11202109972QA (fr)
WO (1) WO2020185867A1 (fr)

Families Citing this family (6)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2021217083A1 (fr) 2020-04-24 2021-10-28 Sorrento Therapeutics, Inc. Récepteurs antigéniques dimères de mémoire
WO2022015956A1 (fr) 2020-07-15 2022-01-20 Sorrento Therapeutics, Inc. Procédé amélioré d'intégration d'adn à l'aide d'endonucléases guidées par arn
WO2022061115A1 (fr) * 2020-09-18 2022-03-24 Vor Biopharma Inc. Compositions et procédés pour modification de cd7
JP2024513087A (ja) * 2021-04-07 2024-03-21 アストラゼネカ・アクチエボラーグ 部位特異的改変のための組成物及び方法
EP4370676A2 (fr) * 2021-06-18 2024-05-22 Artisan Development Labs, Inc. Compositions et procédés de ciblage, d'édition ou de modification de gènes humains
WO2023097236A1 (fr) * 2021-11-24 2023-06-01 The Rockefeller University Compositions et procédés pour générer des souris knock-in d'immunoglobuline

Family Cites Families (12)

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Publication number Priority date Publication date Assignee Title
US5034506A (en) 1985-03-15 1991-07-23 Anti-Gene Development Group Uncharged morpholino-based polymers having achiral intersubunit linkages
US5489677A (en) 1990-07-27 1996-02-06 Isis Pharmaceuticals, Inc. Oligonucleoside linkages containing adjacent oxygen and nitrogen atoms
US5602240A (en) 1990-07-27 1997-02-11 Ciba Geigy Ag. Backbone modified oligonucleotide analogs
US6555674B2 (en) 2000-08-09 2003-04-29 Nsgene A/S JeT promoter
DK3401400T3 (da) 2012-05-25 2019-06-03 Univ California Fremgangsmåder og sammensætninger til rna-styret mål-dna-modifikation og til rna-styret transskriptionsmodulering
US8697359B1 (en) 2012-12-12 2014-04-15 The Broad Institute, Inc. CRISPR-Cas systems and methods for altering expression of gene products
EP3080260B1 (fr) 2013-12-12 2019-03-06 The Broad Institute, Inc. Systèmes crispr-cas et méthodes de modification de l'expression de produits géniques, informations structurales et enzymes cas modulaires inductibles
CN113930455A (zh) * 2014-10-09 2022-01-14 生命技术公司 Crispr寡核苷酸和基因剪辑
JP7030522B2 (ja) * 2015-05-11 2022-03-07 エディタス・メディシン、インコーポレイテッド 幹細胞における遺伝子編集のための最適化ｃｒｉｓｐｒ／ｃａｓ９システムおよび方法
US9790490B2 (en) 2015-06-18 2017-10-17 The Broad Institute Inc. CRISPR enzymes and systems
GB2569733B (en) 2016-09-30 2022-09-14 Univ California RNA-guided nucleic acid modifying enzymes and methods of use thereof
AU2019231315A1 (en) 2018-03-09 2020-09-17 Vivasor, Inc. Dimeric antigen receptors (DAR)

Also Published As

Publication number Publication date
CN113825834A (zh) 2021-12-21
KR20210149734A (ko) 2021-12-09
WO2020185867A1 (fr) 2020-09-17
SG11202109972QA (en) 2021-10-28
US20220145333A1 (en) 2022-05-12
JP2022524435A (ja) 2022-05-02
AU2020239050A1 (en) 2021-11-04
IL286244A (en) 2021-10-31
EP3938510A1 (fr) 2022-01-19
MX2021010938A (es) 2022-01-06

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